Thinking about the FDA? Context Matters

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  • 05/27/2014

Part two of my interview from Context Matters:


In 2010, Representative Henry Waxman voiced concern about non-inferiority trials – but when he was confronted with the science he backed down. That’s a good thing. When politicians start to dabble in science, they generally get badly burned.

Today the FDA is trying to expedite reviews of anti-infectives. The need for these products and that in itself changes the benefit / risk profile. And that will allow for approvals based on less data – and in an expedited fashion.

There are multiple ways to get an important new therapy to market quicker – but that has to be based on a public health need, not a marketing strategy.

Pediatric Trials

Pediatric trials bring both public health and IP benefits, but they are hard to design, hard to recruit for, and hard to field. They’re especially important for anti-depressants and anti-psychotics. We also will be seeing more precise clinical trials in minority and genotype populations. From a perspective of companion diagnostics we’re developing more medicines for small, more precisely identifiable patient populations – clearly the wave of the present and this will continue for the foreseeable future. It’s all about personalized medicine.

The best way to address benefit/risk profile is to make sure that the right patient gets the right medicine in the right dose at the right time. That will result in more positive clinical outcomes and huge cost savings. That’s where you’re going to see the greatest increase in adaptive clinical trials, whether in children, pregnant women, certain genotypes, women, men, African-Americans, Asians, etc.

The benefit/risk profile of a product that’s proven to be more beneficial within an identifiable sub-population is a key for expedited review, because you can more precisely define who it works for and, just as importantly, who should not be using it.

Conditional Approval

As far as so-called “conditional approval” goes, I wouldn’t look for it any time soon in the US. The last time I spoke with senior members of the FDA, I heard comments like, “What does that even mean?” And, then again, do drug developers really want conditional approval? You invest a lot of time and money to get a conditional approval and then the agency decides to take the product off the market? Is that something to roll the dice on?

Unless and until the FDA can ramp up its pharmacovigilance prowess, any kind of provisional approvals will remain problematic. At the moment, the FDA doesn’t have an eye in the sky.

Political Impact

And there’s the issue of Congress politicizing the FDA process. Plan B, Avandia, Zohydro. As Mark McClellan used to say, “If people are saying we’re approving drugs too slowly, and others are saying we’re approving drugs too quickly. We must be doing something right.”

That’s my story… and I’m sticking to it!


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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